CART123 Cells With or Without Ruxolitinib in Relapsed/Refractory Acute Myeloid Leukemia
Phase 1 Trial of Autologous CD123-Directed CAR T-Cells (CART123) as Monotherapy or in Combination With Ruxolitinib in Relapsed/Refractory Acute Myeloid Leukemia
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 0 Years to 29 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Procedures mentioned: Infusions
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "This trial will be conducted at the Children's Hospital of Philadelphia with CART123 infusions occurring in an outpatient setting with close follow-up."
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests how safe CART123 cells are and how well they work, alone or with a drug called ruxolitinib. It is for children and young adults with acute myeloid leukemia (AML), a blood cancer that has come back or has not responded to treatment.
This study is designed to evaluate the safety and effectiveness of CART123 cells either alone or when combined with ruxolitinib in pediatric and young adult subjects with relapsed or refractory AML. Subjects will be enrolled into one of two treatment cohorts: subjects who will receive CART123 alone (Cohort A) or subjects who will receive CART123 in combination with ruxolitinib (Cohort B).
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be age 0–29 for CART123 alone, or 1–29 for CART123 with ruxolitinib. The first person at each dose level with ruxolitinib must be at least 12.
- Have AML that has come back at least twice, come back after a donor stem cell transplant, or stayed after chemotherapy. Tests must show cancer in the bone marrow or outside it, as defined by the study.
- Have a stem cell donor ready so a transplant can happen quickly after CART123 treatment, if needed.
- Meet the study’s kidney, liver, lung, and heart function limits. Some liver test results above the limits may be allowed if AML in the liver caused them.
- Have a score of at least 50 on a scale that measures the ability to do daily activities.
- Agree to use accepted birth control if able to have children.
Who can't join
- Have active hepatitis B or C, which are liver infections.
- Have HIV.
- Have active graft-versus-host disease that needs whole-body treatment. This is when donor cells attack your body.
- Use whole-body steroids or drugs that weaken the immune system during cell collection or infusion, or be likely to need them during collection or after infusion. Certain steroid uses are allowed, including inhaled steroids and hydrocortisone to replace normal hormone levels.
- Have cancer in the brain or spinal cord that is getting worse during treatment, or areas of cancer in this tissue that may raise the risk of harmful effects.
- Be pregnant or breastfeeding.
- Have an active infection that is not controlled.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: * 1\. Age at time of consent: Cohort A: 0-29 years. Cohort B: 1-29 years (Note: the first subject at each dose level of Cohort B must be ≥12 years old) * 2\. Subjects with AML in second or greater relapse, post-transplant relapse, or with chemotherapy-refractory disease. Specifically: 1. Second or greater relapse defined as bone marrow flow cytometric confirmation of myeloid leukemia of at least 0.1% or development of extramedullary disease by imaging or biopsy after second documented complete remission; OR 2. Any detectable disease post-allogeneic transplant with bone marrow flow cytometric confirmation of myeloid leukemia of at least 0.1% or development of extramedullary disease by imaging or biopsy; OR 3. Refractory disease, defined as: Persistent bone marrow involvement with ≥0.1% disease by flow cytometry or persistent extramedullary disease by imaging or biopsy after two courses of induction chemotherapy for patients at initial presentation, ≥ 0.1% bone marrow disease by flow cytometry or persistent extramedullary disease by imaging or biopsy after one course of induction chemotherapy for patients who have relapsed after previously achieving a CR , and ≥ 0.1% bone marrow disease by flow cytometry or persistent extramedullary disease by imaging or biopsy after one course of AML-directed chemotherapy for those with myeloid lineage switch. * 3\. Subjects must have an identified stem cell donor with the ability to proceed rapidly to transplant following CART123 treatment if indicated. * 4\. Adequate organ function defined as: 1. Serum creatinine based on age/gender. 2. Adequate liver function: ALT ≤ 500 U/L, Bilirubin ≤3x the upper limit of normal, and ALT and/or bilirubin results that exceed this range are acceptable if, in the opinion of the physician-investigator (or as confirmed by liver biopsy), the abnormalities are directly related to AML infiltration of the liver. 3. Must have a minimum level of pulmonary reserve defined as ≤Grade 1 dyspnea and \<Grade 3 hypoxia; DLCO ≥ 40% (corrected for anemia if necessary) if PFTs are clinically appropriate as determined by the treating investigator. 4. Left Ventricular Shortening Fraction (LVSF) ≥ 28% or Ejection Fraction (LVEF) ≥ 45% confirmed by echocardiogram or another scan. * 5\. Adequate performance status defined as Lansky or Karnofsky performance score ≥ 50. * 6\. Subjects of reproductive potential must agree to use acceptable birth control methods. Exclusion Criteria: * 1\. Active hepatitis B or active hepatitis C * 2\. HIV infection * 3\. Active acute or chronic GVHD requiring systemic therapy * 4\. Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well. * 5\. CNS disease that is progressive on therapy, or with CNS parenchymal lesions that might increase the risk of CNS toxicity. * 6\. Pregnant or nursing (lactating) subjects. * 7\. Uncontrolled active infection
Study sites (1)
PA
- Children's Hospital of Philadelphia · Philadelphia, PA 19104CART Nurse Navigator · Study contact · 445-942-5891 · cartnursenavigator@chop.eduMelissa Varghese, MS · Study contact · varghesem@chop.eduMore trials in Philadelphia